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Breakthrough in leukaemia treatment

Tamara McLean

New treatments for leukaemia and asthma are in development after Australian scientists discovered a way to stop the production of malfunctioning blood cells.

Researchers at the St Vincent's Institute in Melbourne and Hanson Institute in Adelaide have unravelled the structure of a protein in the blood control system that is key to some blood and inflammatory disorders.

Pharmaceutical company CSL will use their breakthrough to develop new treatments that stop the protein from being activated, hindering cancer spread.

Professor Michael Parker, from the St Vincent's Institute, said his team established the structure of a receptor that controls the actions of a blood-forming regulator called GM-CSF.

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"This regulator has been of interest to researchers and clinicians for many years now because its controller or receptor, found on the surface of blood cells, is critical in regulating their many functions," Prof Parker said.

In leukaemia, an excessive number of malfunctioning white blood cells are produced because some of the signals coming from the receptor are abnormal.

"Because our discovery shows precisely what the receptor looks like and also how it works, we can now begin to design new drugs to rein in the deadly abnormal blood cells," he said.

At present, many leukaemias are treated with chemotherapy that destroys diseased blood cells and bone marrow as well as normal cells.

The researchers hope their work, published in the journal Cell, will lead to targeted therapies, more specific to the malfunctioning cells seen in diseases such as leukaemia.

Drugs which use antibody molecules to disrupt the regulator's work are in development at CSL.

Professor Andrew Roberts, from the Walter and Eliza Hall Institute in Melbourne, said the find was a "major step forward".

"Abnormal GM-CSF signalling is pivotal to a variety of serious and difficult-to-treat diseases," Prof Roberts said.

"With this discovery, rational design of targeted therapies can now be accelerated greatly."